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Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Trace Neuroscience is a biopharmaceutical company dedicated to advancing genetic medicine for patients with neurodegenerative diseases. It specializes in developing targeted antisense oligonucleotide therapies, with its lead product aimed at restoring UNC13A protein function in individuals with Amyotrophic Lateral Sclerosis (ALS) caused by TDP-43 mislocalization. This therapy corrects UNC13A mRNA splicing, potentially improving muscle function and extending survival for these patients.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Enara Bio Limited is a biotechnology company based in Oxford, United Kingdom, specializing in the development of vaccines and immunotherapies for cancer treatment and prevention. Founded in 2016 and previously known as ErVaxx Limited, the company focuses on creating targeted cancer immunotherapies by exploring novel antigens derived from endogenous retroviral DNA sequences. Enara Bio's technology platform enables the identification and assessment of tumor specificity and immunogenic potential, facilitating improved strategies for combating various cancers. Through its innovative approach, the company aims to provide effective therapeutic options for a broad patient population.

Gameto is a biotechnology company focused on advancing female reproductive health through innovative therapies. Recognizing the significant lack of treatment options in this underserved area, Gameto employs cutting-edge scientific techniques to develop solutions that enhance the quality of life for women. The company utilizes cellular engineering to create a platform that produces engineered ovarian and endometrial cell lines capable of mimicking the functions of natural cells, including hormone production and response. This platform underpins a portfolio of cellular therapeutics aimed at addressing various female reproductive diseases. Gameto's first initiative, Fertilo, targets improvements in in vitro fertilization (IVF) and egg freezing processes, striving to make them shorter, safer, and more effective. Additionally, the company is developing Deovo, an organoid model of the female reproductive system, and Ameno, a cell-based therapy designed to mitigate health issues related to primary ovarian insufficiency and menopause. Through these efforts, Gameto aims to transform the landscape of female reproductive health care.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

Tenaya Therapeutics is a preclinical-stage biotechnology company focused on developing therapies for heart disease and heart failure. It operates three product platforms: cellular regeneration, which aims to regenerate heart tissue by reprogramming cardiac fibroblasts into cardiomyocytes via delivery of proprietary transcription factors; gene therapy, which targets cardiac fibroblasts to deliver therapeutic payloads; and precision medicine, which enables personalized approaches to treat heart disease. Founded in 2016 and headquartered in South San Francisco, California, Tenaya seeks to address the underlying cellular pathologies of diseased myocardium with curative therapies that advance from discovery through development.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Rampart Bioscience develops gene medicines to provide long-lasting treatments for a wide range of diseases. It employs proprietary development and delivery platforms to create optimized therapeutics in a revocable, non-viral format, advancing gene-based therapies with improved safety and controllability. The company integrates capabilities from gene delivery, protein sciences, and clinical translation to address genetically driven diseases and translate scientific advances into clinical options.

Adela specializes in developing innovative technologies for early detection of high-morbidity conditions like cancer through routine blood tests. Its proprietary platform efficiently captures and analyzes the methylome, focusing on highly informative regions to enhance diagnostic accuracy.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

NAPIGEN is a biotechnology company focused on advancing genome engineering across a range of organisms, including plants, microbes, and animals. The company's innovative technology specifically targets mitochondria and chloroplasts, which are organelles that contain their own DNA and play crucial roles in cellular energy production. By harnessing the potential of these organelles, NAPIGEN aims to develop solutions that enhance the growth and efficiency of various biological systems, ultimately contributing to a more convenient and sustainable society.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Founded in 2018, Ansa Biotechnologies develops an innovative DNA synthesis technology based on enzymes. This novel approach aims to accelerate biological research and engineering by producing gene-length oligonucleotides more efficiently than traditional chemical methods.

Plexium, Inc. is a biotechnology company based in San Diego, California, specializing in the development of targeted protein degradation therapeutics aimed at treating cancer and neurodegenerative diseases. Founded in 2017, Plexium focuses on identifying E3 ligase modulating small molecules to create innovative therapies. The company has developed a screening platform known as DELPhe, which utilizes advanced techniques to miniaturize cell-based assays for high-content readouts. This platform enables the modulation of E3 ligases to selectively target various cancers, including small cell lung and breast tumors, while also inducing immune responses. By exploring next-generation drug candidates that extend beyond traditional approaches, Plexium is poised to uncover new therapeutic possibilities across a wide range of medical applications.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

Ambagon Therapeutics is a biotechnology company focused on developing small-molecule therapeutics for cancer treatment. The company specializes in research aimed at stabilizing the interactions of oncogenic proteins, which are often difficult to modulate with traditional methods. By selectively enhancing the activity of tumor-suppressing proteins associated with transcription factors and oncoproteins, Ambagon aims to provide new therapeutic options for addressing significant unmet clinical needs in oncology. Founded by Christian Ottmann, Lucas Brunsveld, and Michelle Arkin, the company is dedicated to advancing innovative solutions in cancer care.

Nodexus is a biotech startup focused on providing innovative solutions for live single-cell isolation in the industrial, biopharma, clinical research, and academic sectors. The company commercializes the NX One platform, which features a low-cost, low-infrastructure hardware system and single-use disposable microfluidic cartridges. This platform is designed to enhance workflows related to gene editing, cell line and antibody development, and studies on tumor heterogeneity. By offering affordable automated cell isolation, Nodexus aims to accelerate biological research and make advanced technologies more accessible to a wider range of clients.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that aim to address a wide range of severe medical conditions. By leveraging its innovative technology, Expansion Therapeutics seeks to provide transformative oral treatment options, enhancing the therapeutic landscape for patients suffering from these complex diseases.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Tango Therapeutics is a biotechnology company focused on developing cancer therapies based on the genetic principle of synthetic lethality. It aims to counteract tumor suppressor gene loss, reverse immune evasion in cancer cells, and identify effective drug combinations.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as EngTregs, aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Element Biosciences develops innovative genetic analysis tools for research and diagnostic markets. Founded in 2017, the company specializes in a disruptive DNA sequencing technology that offers high-quality data, workflow flexibility, and affordable access to next-generation sequencing.

Adela specializes in developing innovative technologies for early detection of high-morbidity conditions like cancer through routine blood tests. Its proprietary platform efficiently captures and analyzes the methylome, focusing on highly informative regions to enhance diagnostic accuracy.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Founded in 2018, Scribe Therapeutics specializes in engineering and developing in vivo therapies based on CRISPR technology to rewrite and repair genetic disorders. Its proprietary platform, X-Editing (XE), enables greater efficacy, specificity, and deliverability compared to existing CRISPR technologies.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

Century Therapeutics is a biotechnology company developing allogeneic cell therapies derived from induced pluripotent stem cells to treat cancer and related diseases. Its platform enables engineering of iPSCs into NK and T cells with master cell banks of modified cells that can be expanded and differentiated into standardized, off-the-shelf therapies. The company combines CRISPR-mediated gene editing, proprietary chimeric antigen receptors, Allo-Evasion technology, and manufacturing capabilities to enable scalable production of homogeneous cell products for hematologic and solid tumors. While focused on oncology, its platform also targets autoimmune and inflammatory diseases with the goal of improving cell therapy performance and reducing host immune rejection.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Tenaya Therapeutics is a preclinical-stage biotechnology company focused on developing therapies for heart disease and heart failure. It operates three product platforms: cellular regeneration, which aims to regenerate heart tissue by reprogramming cardiac fibroblasts into cardiomyocytes via delivery of proprietary transcription factors; gene therapy, which targets cardiac fibroblasts to deliver therapeutic payloads; and precision medicine, which enables personalized approaches to treat heart disease. Founded in 2016 and headquartered in South San Francisco, California, Tenaya seeks to address the underlying cellular pathologies of diseased myocardium with curative therapies that advance from discovery through development.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Day One Biopharmaceuticals is a clinical-stage biotechnology company focused on developing targeted therapies for genetically defined cancers across all ages, with an initial emphasis on pediatric patients. The company aims to bring effective cancer treatments to families by advancing therapies that can benefit both children and adults, prioritizing programs that can progress to the market rapidly. Day One pursues licensing or acquisition of promising products from research institutions and industry partners to expand its pipeline. Its lead candidate, DAY101, is an oral, brain-penetrant, highly selective type II pan-RAF kinase inhibitor designed to target cancers driven by the RAF signaling pathway. By applying insights from childhood cancer biology, the company seeks to address unmet needs in oncology and broaden access to precision therapies.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

TScan Therapeutics, Inc. is a biopharmaceutical company dedicated to discovering and developing T cell receptor (TCR) engineered T cell therapies for cancer treatment. Its primary focus is on liquid tumors, with lead therapy candidates TSC-100 and TSC-101 aimed at addressing hematologic malignancies by targeting residual leukemia and preventing relapse following hematopoietic stem cell transplantation. In addition to its liquid tumor programs, TScan is advancing multiplexed TCR-T therapy candidates to tackle various solid tumors. The company employs a unique platform that identifies previously uncharacterized shared T cell antigens and assesses off-target TCR interactions, which supports the creation of TCR-T therapies with reduced off-target effects. Founded in 2018 and headquartered in Waltham, Massachusetts, TScan Therapeutics aims to transform cancer treatment through innovative T cell therapies.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Locanabio is a biotechnology company developing RNA-targeted gene therapies for underserved diseases, with a focus on neuromuscular, neurodegenerative, and retinal conditions. It employs an RNA-targeting platform to modify disease-causing RNA and correct aberrant transcripts, offering therapies distinct from DNA-based approaches. The company identifies target RNA sequences within transcripts and programs therapies to advance candidates for rare genetic neuromuscular, neurodegenerative, and retinal diseases. Located in San Diego, Locanabio aims to translate RNA-directed interventions into treatments for patients with severe conditions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.

Metagenomi is a genetic medicines company headquartered in Emeryville, California. It leverages metagenomics and machine learning to discover novel genome editing systems from diverse organisms, then convert these natural systems into compact, highly efficient, and specific tools aimed at reducing immune responses. The company maintains a proprietary, metagenomics-derived genome editing toolbox that includes programmable nucleases, base editors, small RNA-mediated integration systems (RIGS), large template RIGS, and CRISPR-associated transposases (CASTs). Its development focus is in vivo gene editing with a wholly owned pipeline, while exploring technology out-licensing for ex vivo cell therapies to pursue curative therapeutics for genetic diseases.

AavantiBio, Inc. is a biopharmaceutical company focused on developing gene transfer and gene editing therapies aimed at treating rare genetic diseases. Incorporated in 2019 and based in Gainesville, Florida, the company primarily concentrates on Friedreich’s Ataxia, a rare inherited disorder that leads to significant cardiac and central nervous system dysfunction. AavantiBio is committed to transforming the lives of patients suffering from this condition by advancing innovative therapies that address unmet medical needs in the field of genetic disorders. The company’s efforts are directed toward both treatment development and research, with the goal of providing effective solutions for patients facing the challenges of rare diseases.

Founded in 2020, Be Biopharma specializes in developing innovative therapeutics using engineered B-cells to treat diseases such as cancer, autoimmune disorders, and infectious diseases. Its approach aims to overcome limitations of current cell and gene therapies.

Kinnate Biopharma Inc. is a biopharmaceutical company dedicated to the discovery and development of small molecule kinase inhibitors aimed at treating genomically defined cancers. Established in 2018 and headquartered in San Diego, California, the company focuses on precision oncology therapeutics, leveraging its expertise in structure-based drug discovery and translational research. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is advancing other preclinical candidates, including KIN003, which targets cancer-associated alterations in FGFR2 and FGFR3 genes, and a Cyclin-Dependent Kinase 12 inhibitor in the KIN004 program. Kinnate Biopharma is committed to developing targeted therapeutics for underserved patient populations, aiming to expand the reach of precision medicine in cancer treatment.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as EngTregs, aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

Element Biosciences develops innovative genetic analysis tools for research and diagnostic markets. Founded in 2017, the company specializes in a disruptive DNA sequencing technology that offers high-quality data, workflow flexibility, and affordable access to next-generation sequencing.

Legend Biotech is a clinical-stage biopharmaceutical company focused on discovering and developing novel cell therapies for oncology and other indications. Its lead candidate, LCAR-B38M/JNJ-4528, is an autologous CAR-T therapy for multiple myeloma, evaluated in various trials including earlier-line settings and comparisons with standard therapies in Revlimid-refractory disease. The company also has a broad pipeline of autologous and allogeneic CAR-T and related immunotherapies targeting solid tumors and hematologic cancers, including investigations into CD20-directed allogeneic CAR-T for non-Hodgkin lymphoma in China as an investigator-initiated Phase 1 study, as well as early preclinical and clinical programs for additional cancers and infectious diseases. Legend Biotech was founded in 2014 and is based in Somerset, New Jersey; it is a subsidiary of Genscript Biotech Corporation.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Quench Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing therapies for inflammatory diseases by targeting gasdermin proteins. The company specializes in gasdermin biology and innate immunity to create innovative medicines. Its lead compounds specifically target and inhibit Gasdermin D, a key protein involved in pyroptosis and NETosis pathways, which play significant roles in the release of inflammatory cytokines, alarmins, DNA, and neutrophil extracellular traps (NETs). Established in 2018 and previously known as Project Pyro, Quench Bio aims to provide effective treatments that alleviate the symptoms of severe inflammatory conditions.

Forma Therapeutics, headquartered in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and various cancers. The company is advancing several key product candidates, including FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, targeting metastatic castration-resistant prostate cancer. Additionally, Forma is developing FT-2102, a selective small molecule designed to inhibit mutated IDH1 enzymes, which is being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and in exploratory trials for glioma. Other candidates include FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. The firm integrates advanced drug discovery technologies with deep oncology expertise to address challenging targets, contributing to a robust pipeline aimed at transforming patient outcomes in oncology.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

Enara Bio Limited is a biotechnology company based in Oxford, United Kingdom, specializing in the development of vaccines and immunotherapies for cancer treatment and prevention. Founded in 2016 and previously known as ErVaxx Limited, the company focuses on creating targeted cancer immunotherapies by exploring novel antigens derived from endogenous retroviral DNA sequences. Enara Bio's technology platform enables the identification and assessment of tumor specificity and immunogenic potential, facilitating improved strategies for combating various cancers. Through its innovative approach, the company aims to provide effective therapeutic options for a broad patient population.

Stoke Therapeutics is a biopharmaceutical company developing novel antisense oligonucleotide medicines. These target RNA splicing to increase gene expression, aiming to treat severe genetic diseases. Its lead product candidate, STK-001, focuses on Dravet syndrome, a progressive genetic epilepsy.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

Synthorx, Inc. is a biopharmaceutical company specializing in the development of innovative protein therapeutics using advanced synthetic biology techniques. Founded in 2014 and headquartered in La Jolla, California, Synthorx focuses on its proprietary Synthorin programs aimed at treating cancer and autoimmune disorders. Its lead product candidate, THOR-707, is a modified form of recombinant human IL-2, currently being evaluated for various solid tumors both as a standalone treatment and in combination with immune checkpoint inhibitors. The company also develops Synthorins targeting IL-2 for autoimmune conditions, IL-10 for immuno-oncology applications, and IL-15 for regulating immune responses. Synthorx leverages a unique platform that enables the incorporation of synthetic DNA base pairs to enhance the specificity and efficacy of its protein therapeutics, thus offering improved treatment options for patients. Previously known as Alinos, Inc., the company acquired exclusive rights to its synthetic biology technology based on groundbreaking research from The Scripps Research Institute. As of January 2020, Synthorx operates as a subsidiary of Sanofi.

Eidos Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Francisco, California, specializing in the development of therapies for diseases associated with transthyretin (TTR) amyloidosis (ATTR). Founded in 2013, the company is advancing its lead candidate, AG10, which is currently undergoing phase 3 clinical trials. AG10 is an orally administered small molecule designed to stabilize tetrameric TTR, aiming to interrupt the pathological processes that lead to ATTR. As a subsidiary of BridgeBio Pharma, Eidos Therapeutics is committed to addressing the clinical needs related to TTR and amyloidosis through innovative, genetically targeted therapies.

Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that aim to address a wide range of severe medical conditions. By leveraging its innovative technology, Expansion Therapeutics seeks to provide transformative oral treatment options, enhancing the therapeutic landscape for patients suffering from these complex diseases.

Dyne Therapeutics is a clinical-stage biotechnology company developing therapies for genetically driven neuromuscular diseases. It aims to deliver disease-modifying therapeutics to skeletal, cardiac, and smooth muscle, addressing conditions such as muscular dystrophies and other muscle disorders. The company uses the FORCE platform to create targeted therapeutics with payloads designed to address the disease gene, connected via a linker to an antigen-binding fragment, enabling delivery to muscle tissue and the central nervous system. Its pipeline includes DYNE-101, DYNE-251, DYNE-302, and DYNE-401. Headquartered in Waltham, Massachusetts.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

Synthorx, Inc. is a biopharmaceutical company specializing in the development of innovative protein therapeutics using advanced synthetic biology techniques. Founded in 2014 and headquartered in La Jolla, California, Synthorx focuses on its proprietary Synthorin programs aimed at treating cancer and autoimmune disorders. Its lead product candidate, THOR-707, is a modified form of recombinant human IL-2, currently being evaluated for various solid tumors both as a standalone treatment and in combination with immune checkpoint inhibitors. The company also develops Synthorins targeting IL-2 for autoimmune conditions, IL-10 for immuno-oncology applications, and IL-15 for regulating immune responses. Synthorx leverages a unique platform that enables the incorporation of synthetic DNA base pairs to enhance the specificity and efficacy of its protein therapeutics, thus offering improved treatment options for patients. Previously known as Alinos, Inc., the company acquired exclusive rights to its synthetic biology technology based on groundbreaking research from The Scripps Research Institute. As of January 2020, Synthorx operates as a subsidiary of Sanofi.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

Calimmune is a clinical-stage gene therapy company focused on HIV, developing cell-based therapies to protect individuals with HIV from progression to AIDS. Its lead candidate, Cal-1, is a gene-based therapy designed to modify immune cells to reduce HIV entry and replication by lowering CCR5 and preventing viral fusion, with activity demonstrated in preclinical studies and evaluated in Phase I/II trials. The company is based in Tucson, Arizona, and aims to address HIV at various stages of infection and treatment history by delivering durable cellular therapies that strengthen the immune system.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

Dimension Therapeutics, Inc. is a gene therapy company based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

Natera, Inc. is a diagnostic and research company specializing in genetic testing services, particularly in the areas of preconception and prenatal diagnostics. Founded in 2003 and headquartered in San Carlos, California, Natera offers a range of products including the Panorama non-invasive prenatal test, which screens for chromosomal abnormalities in fetuses, and Vistara, a test for single-gene mutations. Additionally, the company provides Horizon carrier screening to assess carrier status for various genetic diseases, and Spectrum, which analyzes genetic conditions during in vitro fertilization. Natera's Anora product analyzes fetal chromosomes to understand miscarriage causes, while its non-invasive paternity tests determine parentage through fetal DNA analysis. The company's Signatera technology focuses on circulating tumor DNA to monitor cancer recurrence. Natera distributes its products through a direct sales force and a network of approximately 100 laboratory and distribution partners, both domestically and internationally. The company collaborates with BGI Genomics to develop genetic testing assays and has partnered with Foundation Medicine for personalized cancer monitoring assays.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

Nivalis Therapeutics is a biotechnology company focused on developing innovative disease-modifying therapies aimed at cystic fibrosis (CF). The company is particularly dedicated to preserving intracellular S-nitrosoglutathione (GSNO), a naturally occurring molecule that plays a crucial role in cell signaling and is linked to the pathophysiology of CF. Its lead therapeutic candidate, N91115, specifically targets patients with the F508del mutation, which is the most prevalent mutation responsible for cystic fibrosis. Founded in 2007 and based in Boulder, Colorado, Nivalis Therapeutics aims to significantly improve the quality of life for individuals affected by cystic fibrosis and their families.

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Arrowhead Pharmaceuticals is a biotechnology company focused on creating medicines for diseases with a genetic basis driven by overproduction of proteins. The company develops RNA interference therapeutics designed to silence disease-related genes and reduce pathogenic protein production. Its pipeline includes candidates such as ARO-AAT for liver disease caused by alpha-1 antitrypsin deficiency, ARO-APOC3 for high triglycerides, ARO-ANG3 to lower angiopoietin-like protein 3, ARO-HSD for liver disorders, ARO-ENaC to modulate airway sodium channels, ARO-Lung2 for chronic obstructive pulmonary disease, and ARO-COV for pulmonary infections, with ARO-HIF2 in earlier stage for certain kidney cancer. It is also pursuing programs like JNJ-3989 for chronic hepatitis B, Olpasiran for apolipoprotein A production, and ARO-AMG1 for cardiovascular targets. The company collaborates with Janssen and Takeda on RNAi therapeutics and was formerly Arrowhead Research Corporation, renamed Arrowhead Pharmaceuticals in 2016 after being established in 1989. It is headquartered in Pasadena, California.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.